Genome engineering of stem cell organoids for disease modeling.

Abstract:

:Precision medicine emerges as a new approach that takes into account individual variability. Successful realization of precision medicine requires disease models that are able to incorporate personalized disease information and recapitulate disease development processes at the molecular, cellular and organ levels. With recent development in stem cell field, a variety of tissue organoids can be derived from patient specific pluripotent stem cells and adult stem cells. In combination with the state-of-the-art genome editing tools, organoids can be further engineered to mimic disease-relevant genetic and epigenetic status of a patient. This has therefore enabled a rapid expansion of sophisticated in vitro disease models, offering a unique system for fundamental and biomedical research as well as the development of personalized medicine. Here we summarize some of the latest advances and future perspectives in engineering stem cell organoids for human disease modeling.

journal_name

Protein Cell

journal_title

Protein & cell

authors

Sun Y,Ding Q

doi

10.1007/s13238-016-0368-0

subject

Has Abstract

pub_date

2017-05-01 00:00:00

pages

315-327

issue

5

eissn

1674-800X

issn

1674-8018

pii

10.1007/s13238-016-0368-0

journal_volume

8

pub_type

杂志文章,评审
  • Domesticated cynomolgus monkey embryonic stem cells allow the generation of neonatal interspecies chimeric pigs.

    abstract::Blastocyst complementation by pluripotent stem cell (PSC) injection is believed to be the most promising method to generate xenogeneic organs. However, ethical issues prevent the study of human chimeras in the late embryonic stage of development. Primate embryonic stem cells (ESCs), which have similar pluripotency to ...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-019-00676-8

    authors: Fu R,Yu D,Ren J,Li C,Wang J,Feng G,Wang X,Wan H,Li T,Wang L,Zhang Y,Hai T,Li W,Zhou Q

    更新日期:2020-02-01 00:00:00

  • Trafficking abnormality and ER stress underlie functional deficiency of hearing impairment-associated connexin-31 mutants.

    abstract::Hearing impairment (HI) affects 1/1000 children and over 2% of the aged population. We have previously reported that mutations in the gene encoding gap junction protein connexin-31 (C×31) are associated with HI. The pathological mechanism of the disease mutations remains unknown. Here, we show that expression of C×31 ...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-010-0118-7

    authors: Xia K,Ma H,Xiong H,Pan Q,Huang L,Wang D,Zhang Z

    更新日期:2010-10-01 00:00:00

  • Dephosphorylation of cGAS by PPP6C impairs its substrate binding activity and innate antiviral response.

    abstract::The cyclic GMP-AMP (cGAMP) synthase (cGAS) plays a critical role in host defense by sensing cytosolic DNA derived from microbial pathogens or mis-located cellular DNA. Upon DNA binding, cGAS utilizes GTP and ATP as substrates to synthesize cGAMP, leading to MITA-mediated innate immune response. In this study, we ident...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-020-00729-3

    authors: Li M,Shu HB

    更新日期:2020-08-01 00:00:00

  • IgG Fc engineering to modulate antibody effector functions.

    abstract::Therapeutic monoclonal antibodies are among the most effective biotherapeutics to date. An important aspect of antibodies is their ability to bind antigen while at the same time recruit immune effector functions. The majority of approved recombinant monoclonal antibody therapies are of the human IgG1 subclass, which c...

    journal_title:Protein & cell

    pub_type: 杂志文章,评审

    doi:10.1007/s13238-017-0473-8

    authors: Wang X,Mathieu M,Brezski RJ

    更新日期:2018-01-01 00:00:00

  • MicroRNAs: a new ray of hope for diabetes mellitus.

    abstract::Diabetes mellitus has become one of the most common chronic diseases, thereby posing a major challenge to global health. Characterized by high levels of blood glucose (hyperglycemia), diabetes usually results from a loss of insulin-producing β-cells in the pancreas, leading to a deficiency of insulin (type 1 diabetes)...

    journal_title:Protein & cell

    pub_type: 杂志文章,评审

    doi:10.1007/s13238-012-2055-0

    authors: Kumar M,Nath S,Prasad HK,Sharma GD,Li Y

    更新日期:2012-10-01 00:00:00

  • SNObase, a database for S-nitrosation modification.

    abstract::S-Nitros(yl)ation is a ubiquitous redox-based post-translational modification of protein cysteine thiols by nitric oxide or its derivatives, which transduces the bioactivity of nitric oxide (NO) by regulation of protein conformation, activity, stability, localization and protein-protein interactions. These years, more...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-012-2094-6

    authors: Zhang X,Huang B,Zhang L,Zhang Y,Zhao Y,Guo X,Qiao X,Chen C

    更新日期:2012-12-01 00:00:00

  • Mechanism involved in the modulation of photoreceptor-specific cyclic nucleotidegated channel by the tyrosine kinase adapter protein Grb14.

    abstract::We recently found that growth factor receptor-bound (Grb) protein 14 is a novel physiological modulator of photoreceptor specific cyclic nucleotide-gated channel alpha subunit (CNGA1). Grb14 promotes the CNG channel closure through its Ras-associating (RA) domain. In the current study we show that this RA domain-media...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-011-1115-1

    authors: Gupta VK,Rajala A,Rodgers KK,Rajala RV

    更新日期:2011-11-01 00:00:00

  • Directed hepatic differentiation from embryonic stem cells.

    abstract::The liver is the largest internal organ in mammals, and is important for the maintenance of normal physiological functions of other tissues and organs. Hepatitis, cirrhosis, liver cancer and other chronic liver diseases are serious threats to human health, and these problems are compounded by a scarcity of liver donor...

    journal_title:Protein & cell

    pub_type: 杂志文章,评审

    doi:10.1007/s13238-011-1023-4

    authors: Chen X,Zeng F

    更新日期:2011-03-01 00:00:00

  • Functional regulation of monocyte-derived dendritic cells by microRNAs.

    abstract::Dendritic cells (DCs) as a rare type of leukocytes play an important role in bridging the innate and adaptive immune system. A subset of DCs, monocyte-derived dendritic cells (moDCs), exists in very low numbers at steady state but become abundant in inflammatory states. These inflammation-associated DCs are potent pro...

    journal_title:Protein & cell

    pub_type: 杂志文章,评审

    doi:10.1007/s13238-012-0042-0

    authors: Zhan Y,Wu L

    更新日期:2012-07-01 00:00:00

  • Dscam mutation leads to hydrocephalus and decreased motor function.

    abstract::The nervous system is one of the most complicated organ systems in invertebrates and vertebrates. Down syndrome cell adhesion molecule (DSCAM) of the immunoglobulin (Ig) superfamily is expressed widely in the nervous system during embryonic development. Previous studies in Drosophila suggest that Dscam plays important...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-011-1072-8

    authors: Xu Y,Ye H,Shen Y,Xu Q,Zhu L,Liu J,Wu JY

    更新日期:2011-08-01 00:00:00

  • Proteomic identification and functional characterization of MYH9, Hsc70, and DNAJA1 as novel substrates of HDAC6 deacetylase activity.

    abstract::Histone deacetylase 6 (HDAC6), a predominantly cytoplasmic protein deacetylase, participates in a wide range of cellular processes through its deacetylase activity. However, the diverse functions of HDAC6 cannot be fully elucidated with its known substrates. In an attempt to explore the substrate diversity of HDAC6, w...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-014-0102-8

    authors: Zhang L,Liu S,Liu N,Zhang Y,Liu M,Li D,Seto E,Yao TP,Shui W,Zhou J

    更新日期:2015-01-01 00:00:00

  • Complement activation by phospholipids: the interplay of factor H and C1q.

    abstract::Complement proteins in blood recognize charged particles. The anionic phospholipid (aPL) cardiolipin binds both complement proteins C1q and factor H. C1q is an activator of the complement classical pathway, while factor H is an inhibitor of the alternative pathway. To examine opposing effects of C1q and factor H on co...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-010-0125-8

    authors: Tan LA,Yu B,Sim FC,Kishore U,Sim RB

    更新日期:2010-11-01 00:00:00

  • System approaches reveal the molecular networks involved in neural stem cell differentiation.

    abstract::The self-renewal and multipotent potentials in neural stem cells (NSCs) maintain the normal physiological functions of central nervous system (CNS). The abnormal differentiation of NSCs would lead to CNS disorders. However, the mechanisms of how NSCs differentiate into astrocytes, oligodendrocytes (OLs) and neurons ar...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-012-0014-4

    authors: Wang K,Wang H,Wang J,Xie Y,Chen J,Yan H,Liu Z,Wen T

    更新日期:2012-03-01 00:00:00

  • Atg7 in development and disease: panacea or Pandora's Box?

    abstract::Macroautophagy is an evolutionarily conserved intracellular degradation system used by life ranging from yeasts to mammals. The core autophagic machinery is composed of ATG (autophagy-related) protein constituents. One particular member of the ATG protein family, Atg7, has been the focus of recent research. Atg7 acts ...

    journal_title:Protein & cell

    pub_type: 杂志文章,评审

    doi:10.1007/s13238-015-0195-8

    authors: Xiong J

    更新日期:2015-10-01 00:00:00

  • Structural dynamics of the yeast Shwachman-Diamond syndrome protein (Sdo1) on the ribosome and its implication in the 60S subunit maturation.

    abstract::The human Shwachman-Diamond syndrome (SDS) is an autosomal recessive disease caused by mutations in a highly conserved ribosome assembly factor SBDS. The functional role of SBDS is to cooperate with another assembly factor, elongation factor 1-like (Efl1), to promote the release of eukaryotic initiation factor 6 (eIF6...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-015-0242-5

    authors: Ma C,Yan K,Tan D,Li N,Zhang Y,Yuan Y,Li Z,Dong MQ,Lei J,Gao N

    更新日期:2016-03-01 00:00:00

  • POST1/C12ORF49 regulates the SREBP pathway by promoting site-1 protease maturation.

    abstract::Sterol-regulatory element binding proteins (SREBPs) are the key transcriptional regulators of lipid metabolism. The activation of SREBP requires translocation of the SREBP precursor from the endoplasmic reticulum to the Golgi, where it is sequentially cleaved by site-1 protease (S1P) and site-2 protease and releases a...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-020-00753-3

    authors: Xiao J,Xiong Y,Yang LT,Wang JQ,Zhou ZM,Dong LW,Shi XJ,Zhao X,Luo J,Song BL

    更新日期:2020-07-14 00:00:00

  • Modeling CADASIL vascular pathologies with patient-derived induced pluripotent stem cells.

    abstract::Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL) is a rare hereditary cerebrovascular disease caused by a NOTCH3 mutation. However, the underlying cellular and molecular mechanisms remain unidentified. Here, we generated non-integrative induced pluripotent stem cells...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-019-0608-1

    authors: Ling C,Liu Z,Song M,Zhang W,Wang S,Liu X,Ma S,Sun S,Fu L,Chu Q,Belmonte JCI,Wang Z,Qu J,Yuan Y,Liu GH

    更新日期:2019-04-01 00:00:00

  • Structures of the N- and C-terminal domains of MHV-A59 nucleocapsid protein corroborate a conserved RNA-protein binding mechanism in coronavirus.

    abstract::Coronaviruses are the causative agent of respiratory and enteric diseases in animals and humans. One example is SARS, which caused a worldwide health threat in 2003. In coronaviruses, the structural protein N (nucleocapsid protein) associates with the viral RNA to form the filamentous nucleocapsid and plays a crucial ...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-010-0079-x

    authors: Ma Y,Tong X,Xu X,Li X,Lou Z,Rao Z

    更新日期:2010-07-01 00:00:00

  • Proteomic and transcriptomic analysis of visual long-term memory in Drosophila melanogaster.

    abstract::The fruit fly, Drosophila melanogaster, is able to discriminate visual landmarks and form visual long-term memory in a flight simulator. Studies focused on the molecular mechanism of long-term memory have shown that memory formation requires mRNA transcription and protein synthesis. However, little is known about the ...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-011-1019-0

    authors: Jiang H,Hou Q,Gong Z,Liu L

    更新日期:2011-03-01 00:00:00

  • Extracellular signal regulated kinase 5 promotes cell migration, invasion and lung metastasis in a FAK-dependent manner.

    abstract::This study was designed to evaluate ERK5 expression in lung cancer and malignant melanoma progression and to ascertain the involvement of ERK5 signaling in lung cancer and melanoma. We show that ERK5 expression is abundant in human lung cancer samples, and elevated ERK5 expression in lung cancer was linked to the acqu...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-020-00701-1

    authors: Jiang W,Cai F,Xu H,Lu Y,Chen J,Liu J,Cao N,Zhang X,Chen X,Huang Q,Zhuang H,Hua ZC

    更新日期:2020-11-01 00:00:00

  • Molecular basis for the inhibition of β-hydroxyacyl-ACP dehydratase HadAB complex from Mycobacterium tuberculosis by flavonoid inhibitors.

    abstract::Dehydration is one of the key steps in the biosynthesis of mycolic acids and is vital to the growth of Mycobacterium tuberculosis (Mtb). Consequently, stalling dehydration cures tuberculosis (TB). Clinically used anti-TB drugs like thiacetazone (TAC) and isoxyl (ISO) as well as flavonoids inhibit the enzyme activity o...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-015-0181-1

    authors: Dong Y,Qiu X,Shaw N,Xu Y,Sun Y,Li X,Li J,Rao Z

    更新日期:2015-07-01 00:00:00

  • Binding of Shewanella FadR to the fabA fatty acid biosynthetic gene: implications for contraction of the fad regulon.

    abstract::The Escherichia coli fadR protein product, a paradigm/prototypical FadR regulator, positively regulates fabA and fabB, the two critical genes for unsaturated fatty acid (UFA) biosynthesis. However the scenario in the other Ɣ-proteobacteria, such as Shewanella with the marine origin, is unusual in that Rodionov and cow...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-015-0172-2

    authors: Zhang H,Zheng B,Gao R,Feng Y

    更新日期:2015-09-01 00:00:00

  • Wld(S), Nmnats and axon degeneration--progress in the past two decades.

    abstract::A chimeric protein called Wallerian degeneration slow (Wld(S)) was first discovered in a spontaneous mutant strain of mice that exhibited delayed Wallerian degeneration. This provides a useful tool in elucidating the mechanisms of axon degeneration. Over-expression of Wld(S) attenuates the axon degeneration that is as...

    journal_title:Protein & cell

    pub_type: 杂志文章,评审

    doi:10.1007/s13238-010-0021-2

    authors: Feng Y,Yan T,He Z,Zhai Q

    更新日期:2010-03-01 00:00:00

  • Novel and potent inhibitors targeting DHODH are broad-spectrum antivirals against RNA viruses including newly-emerged coronavirus SARS-CoV-2.

    abstract::Emerging and re-emerging RNA viruses occasionally cause epidemics and pandemics worldwide, such as the on-going outbreak of the novel coronavirus SARS-CoV-2. Herein, we identified two potent inhibitors of human DHODH, S312 and S416, with favorable drug-likeness and pharmacokinetic profiles, which all showed broad-spec...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-020-00768-w

    authors: Xiong R,Zhang L,Li S,Sun Y,Ding M,Wang Y,Zhao Y,Wu Y,Shang W,Jiang X,Shan J,Shen Z,Tong Y,Xu L,Chen Y,Liu Y,Zou G,Lavillete D,Zhao Z,Wang R,Zhu L,Xiao G,Lan K,Li H,Xu K

    更新日期:2020-10-01 00:00:00

  • The evolving landscape in the therapy of acute myeloid leukemia.

    abstract::Acute myeloid leukemia (AML) is a heterogeneous clonal disorder of myeloid precursors arrested in their maturation, creating a diverse disease entity with a wide range of responses to historically standard treatment approaches. While significant progress has been made in characterizing and individualizing the disease ...

    journal_title:Protein & cell

    pub_type: 杂志文章,评审

    doi:10.1007/s13238-013-3057-2

    authors: Peloquin GL,Chen YB,Fathi AT

    更新日期:2013-10-01 00:00:00

  • Crystal structure of a novel non-Pfam protein PF2046 solved using low resolution B-factor sharpening and multi-crystal averaging methods.

    abstract::Sometimes crystals cannot diffract X-rays beyond 3.0 Å resolution due to the intrinsic flexibility associated with the protein. Low resolution diffraction data not only pose a challenge to structure determination, but also hamper interpretation of mechanistic details. Crystals of a 25.6 kDa non-Pfam, hypothetical prot...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-010-0045-7

    authors: Su J,Li Y,Shaw N,Zhou W,Zhang M,Xu H,Wang BC,Liu ZJ

    更新日期:2010-05-01 00:00:00

  • A novel xeno-free and feeder-cell-free system for human pluripotent stem cell culture.

    abstract::While human induced pluripotent stem cells (hiPSCs) have promising applications in regenerative medicine, most of the hiPSC lines available today are not suitable for clinical applications due to contamination with nonhuman materials, such as sialic acid, and potential pathogens from animal-product-containing cell cul...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-012-2002-0

    authors: Wang Q,Mou X,Cao H,Meng Q,Ma Y,Han P,Jiang J,Zhang H,Ma Y

    更新日期:2012-01-01 00:00:00

  • Human BDCA2+CD123+CD56+ dendritic cells (DCs) related to blastic plasmacytoid dendritic cell neoplasm represent a unique myeloid DC subset.

    abstract::Dendritic cells (DCs) comprise two functionally distinct subsets: plasmacytoid DCs (pDCs) and myeloid DCs (mDCs). pDCs are specialized in rapid and massive secretion of type I interferon (IFN-I) in response to nucleic acids through Toll like receptor (TLR)-7 or TLR-9. In this report, we characterized a CD56(+) DC popu...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-015-0140-x

    authors: Yu H,Zhang P,Yin X,Yin Z,Shi Q,Cui Y,Liu G,Wang S,Piccaluga PP,Jiang T,Zhang L

    更新日期:2015-04-01 00:00:00

  • Effect of the microbial lipopeptide on tumor cell lines: apoptosis induced by disturbing the fatty acid composition of cell membrane.

    abstract::Microbial lipopeptides play an important role in apoptosis induction of tumor cells. However, there is little knowledge about the relationship between apoptosis induction and membrane fatty acids. The present study focused on the effects of lipopeptides produced by Bacillus subtilis HSO121 on Bcap-37 cell lines. 3-(4,...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-010-0072-4

    authors: Liu X,Tao X,Zou A,Yang S,Zhang L,Mu B

    更新日期:2010-06-01 00:00:00

  • Crystal structures of the two membrane-proximal Ig-like domains (D3D4) of LILRB1/B2: alternative models for their involvement in peptide-HLA binding.

    abstract::Leukocyte immunoglobulin-like receptors (LILRs), also called CD85s, ILTs, or LIRs, are important mediators of immune activation and tolerance that contain tandem immunoglobulin (Ig)-like folds. There are 11 (in addition to two pseudogenes) LILRs in total, two with two Ig-like domains (D1D2) and the remaining nine with...

    journal_title:Protein & cell

    pub_type: 杂志文章

    doi:10.1007/s13238-013-3908-x

    authors: Nam G,Shi Y,Ryu M,Wang Q,Song H,Liu J,Yan J,Qi J,Gao GF

    更新日期:2013-10-01 00:00:00